Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: The Primary Immune Deficiency Treatment Consortium experience Academic Article uri icon

Overview

MeSH Major

  • Severe Combined Immunodeficiency

abstract

  • Lack of complete laboratory evaluation of patients before HCT presents a significant barrier to definitive diagnosis of SCID and related disorders and prevented inclusion of subjects in our observational HCT study. This lesson is critical for patient care, as well as the design of future prospective treatment studies for such children because a well-defined and consistent study population is important for precision in outcomes analysis.

publication date

  • January 2014

Research

keywords

  • Academic Article

Identity

Language

  • eng

PubMed Central ID

  • PMC3972266

Digital Object Identifier (DOI)

  • 10.1016/j.jaci.2013.09.044

PubMed ID

  • 24290292

Additional Document Info

start page

  • 1092

end page

  • 8

volume

  • 133

number

  • 4